EAST HANOVER, N.J., Sept. 16, 2011 /PRNewswire/ -- Novartis
announced today positive results of the first pivotal Phase III
trial of ACZ885 in patients with systemic juvenile idiopathic
arthritis (SJIA), a rare and serious childhood auto-inflammatory
disease[3]. The results, presented at the 2011 European Pediatric
Rheumatology Congress in Bruges, Belgium, showed all primary and
secondary endpoints of the study were met[2].

Most ACZ885 patients (83.7%) experienced at least a 30%
improvement in symptoms vs. 9.8% for placebo (p<0.0001) and a
third of ACZ885 patients (32.6%) achieved a 100% improvement vs. 0%
for placebo (p=0.0001)[2]. ACZ885 is an investigational, fully
human monoclonal antibody that neutralizes interleukin-1 beta (IL-1
beta), which is a key driver of inflammation in SJIA[1].

"SJIA is the most severe form of juvenile arthritis. Many of my
patients suffer terribly from this disease, resulting in a critical
need for new treatment options," said Daniel Lovell, M.D., one of
the study investigators and Professor of Pediatrics at the
Cincinnati Children's Hospital Medical Center. "This study showed
ACZ885 effectively relieved the systemic and arthritic disease
components evaluated in the trial, demonstrating a much-needed
benefit for this patient population."

SJIA affects less than one child per 100,000 worldwide[5]. It is
called 'systemic' because the inflammation affects the whole body,
as well as most of the joints. The condition is characterized by
potentially life-long and recurrent arthritis flares, which can
involve skin rash, daily spiking fever, joint pain and
swelling[3,4].

In this study, patients were evaluated according to the adapted
American College of Rheumatology (ACR) Pediatric criteria, which
includes absence of fever. The ACR criteria are regularly used to
assess the success of treatments in SJIA.

"These results are a positive development for patients suffering
from this very severe auto-inflammatory condition," said David
Epstein, Head of the Pharmaceuticals Division of Novartis. "We are
committed to investigate ACZ885 in a range of inflammatory diseases
where interleukin-1 beta plays a key role and high unmet medical
needs exist."

The results of a second pivotal Phase III trial, aimed at
determining whether ACZ885 can extend the time to next flare and
reduce or eliminate corticosteroid use, will be presented later
this year. Worldwide regulatory submissions for ACZ885 in SJIA are
planned for 2012.

About the Study

The study was a Phase III, 4-week, randomized, double-blind,
placebo-controlled study involving 84 patients between the ages of
2 and 19 years, with active SJIA[2]. Patients were treated with
either a single subcutaneous (s.c.) dose of ACZ885 (4 mg/kg, up to
300 mg) or placebo[2].

The primary endpoint was the proportion of patients achieving
the adapted ACR Pediatric 30 criteria, demonstrating a 30%
improvement from baseline at Day 15 in at least three of the six
variables[2]. The six variables were physician's assessment of
disease activity, parent's or patient's assessment of overall
well-being, functional ability, number of joints with active
arthritis, number of joints with limitation of motion and
C-reactive protein, a laboratory measure of inflammation[2]. Body
temperature also was measured[2].

Secondary endpoints included the proportion of patients
achieving the adapted ACR Pediatric 50, 70, 90 or 100 criteria,
demonstrating a 50%, 70%, 90% or 100% improvement in at least three
variables from baseline at Day 15 or 29[2].

ACZ885 was generally well tolerated. During the study, 55.8% of
patients experienced adverse events (AEs), including infections,
with ACZ885 vs. 39% with placebo[2]. Serious adverse events (SAEs),
including infections, were reported for two patients for ACZ885 vs.
two for placebo[2]. These did not lead to discontinuation and were
resolved without complications[2].

About ACZ885

ACZ885 is a fully human monoclonal antibody that inhibits IL-1
beta, which is an important part of the body's immune system
defenses[1]. Excessive production of IL-1 beta plays a major role
in many inflammatory diseases, including SJIA[6]. ACZ885 works by
neutralizing IL-1 beta for a sustained period of time, therefore
inhibiting inflammation[1].

ACZ885 is currently approved in the US and other countries for a
different disease state.

Disclaimer

The foregoing release contains forward-looking statements that
can be identified by terminology such as "planned," "potentially,"
"committed," "will," or similar expressions, or by express or
implied discussions regarding potential new indications or labeling
for ACZ885 or regarding potential future revenues from ACZ885. You
should not place undue reliance on these statements. Such
forward-looking statements reflect the current views of management
regarding future events, and involve known and unknown risks,
uncertainties and other factors that may cause actual results with
ACZ885 to be materially different from any future results,
performance or achievements expressed or implied by such
statements. There can be no guarantee that ACZ885 will be approved
for any additional indications or labeling in any market. Nor can
there be any guarantee that ACZ885 will achieve any particular
levels of revenue in the future. In particular, management's
expectations regarding ACZ885 could be affected by, among other
things, unexpected clinical trial results, including unexpected new
clinical data and unexpected additional analysis of existing
clinical data; unexpected regulatory actions or delays or
government regulation generally; competition in general;
government, industry and general public pricing pressures; the
company's ability to obtain or maintain patent or other proprietary
intellectual property protection; the impact that the foregoing
factors could have on the values attributed to the Novartis Group's
assets and liabilities as recorded in the Group's consolidated
balance sheet, and other risks and factors referred to in Novartis
AG's current Form 20-F on file with the US Securities and Exchange
Commission. Should one or more of these risks or uncertainties
materialize, or should underlying assumptions prove incorrect,
actual results may vary materially from those anticipated,
believed, estimated or expected. Novartis is providing the
information in this press release as of this date and does not
undertake any obligation to update any forward-looking statements
contained in this press release as a result of new information,
future events or otherwise.

Located in East Hanover, New Jersey, Novartis Pharmaceuticals
Corporation is an affiliate of Novartis AG, which provides
healthcare solutions that address the evolving needs of patients
and societies. Focused solely on healthcare, Novartis offers a
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is the only company with leading positions in these areas. In 2010,
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excluding impairment and amortization charges) was invested in
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